Gene editing technologies allow the researchers to change the genetic material of an organism. This change can be due to any addition, deletion or alt
Gene editing technologies allow the researchers to change the genetic material of an organism. This change can be due to any addition, deletion or alteration at specific location in the genome. Basically, there are three ways in which genes can be manipulated, namely:[1]
- Gene Insertion: This involves the addition of new attributes to a gene through the incorporation of nucleotide sequences.
- Gene Repair: This refers to the replacement of a defective gene sequence by a functional sequence.
- Gene Inactivation: This involves the use of specific nucleotide sequences or regulatory elements to prevent the expression of a target gene.
Table 10.1 provides information on the companies that are using / have developed proprietary gene editing technology platforms that can be applied for the production of gene therapies.
Table 10.1 Gene Editing Technology Platforms
| S. No. | Company Name | HQ | Technology | Number of Molecules in Pipeline[2] | Highest Phase of Development |
| 1 | Beam Therapeutics | US | CRISPR[3] | 1 | Undisclosed |
| 2 | Bioverativ, Sangamo Therapeutics | US | ZFN Technology[4] | 1 | I/II |
| 3 | bluebird bio | US | Homing endonuclease and megaTAL[5] | 1 | Preclinical |
| 4 | Caribou Biosciences | US | CRISPR / Cas[6] | NA | NA |
| 5 | Cellectis | France | TALEN[7], [8] | NA | NA |
| 6 | CRISPR Therapeutics | US | CRISPR / Cas9[9] | 5 | I/II |
| 7 | EdiGene | China | CRISPR[10] | 4 | I/II (Planned) |
| 8 | Editas Medicine | US | CRISPR / Cas9, TALEN[11], [12] | 6 | I/II |
| 9 | Exonics Therapeutics | US | CRISPR / Cas9[13] | 1 | Preclinical |
| 10 | Homology Medicines | US | Proprietary Technology[14] | 4 | Preclinical |
| 11 | Horizon Discovery Group | England | Proprietary Technology[15] | NA | NA |
| 12 | Intellia Therapeutics | US | CRISPR / Cas9[16] | 5 | Preclinical |
| 13 | LogicBio Therapeutics | US | GeneRide[17], [18] | 4 | Preclinical |
| 14 | Novartis | Switzerland | Proprietary AAV-mediated editing by Directed Homologous Recombination[19] | NA | NA |
| 15 | Poseida Therapeutics | US | Cas-CLOVER[20] | NA | NA |
| 16 | Precision BioSciences | US | ARCUS[21], [22] | NA | NA |
| 17 | Sangamo Therapeutics | US | ZFN Technology[23] | 5 | I/II |
| 18 | Sarepta Therapeutics, Duke University | US | CRISPR / Cas9[24] | NA | Discovery |
| 19 | Transposagen | US | Footprint-Free[25], [26] | NA | NA |
| 20 | Verve Therapeutics | US | CRISPR[27] | 1 | Preclinical |
Abbreviations: HQ: Headquarters; ZFN: Zinc Finger Nuclease; TALEN: Transcription Activator-like Effector Nuclease
Source: Roots Analysis
COMMENTS